How is real-world evidence transforming the drug development paradigm? 14 Apr 2021
Q&A with Dr Sheuli Porkess
Real-world data and real-world evidence are transforming the drug development paradigm. Over the last 12 months, our use of patient data has been more vital than ever to support public health and gain information about vital medicines. In this article, we interview Dr Sheuli Porkess for her insights on the latest trends and her expectations for the future.
What are real-world data and real-world evidence?
The FDA defines real-world data as ‘data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources.’ FDA’s examples of real-world data, collected in daily life, include health records, product registries, and data from wearables.
It’s a broad definition that will only expand further as technology allows for more data collection. The next step is turning that information into insights that give us the answer to a question – becoming real-world evidence. Data science helps us collect, process, and analyse the data appropriately to draw robust conclusions while ensuring data privacy.
The use of real-world data is driven not only by technology but by policy decisions about how individual countries assemble data about their citizens.
The pharmaceutical industry is keenly interested in this area. It is a marker of interest that associations such as the ABPI and EFPIA pursue the topic in their roadmaps. For example, the EFPIA Regulatory Road to Innovation paper devotes a section to real-world data and real-world evidence. It recommends that EMA develop and adopt a real-world evidence framework with clear principles for data quality, interoperability, access analysis, and regulatory acceptance.
Regulatory acceptance is critical as sponsors want to ensure the evidence they present is valuable and will support approval and reimbursement. Agencies including the FDA and MHRA have worked intensively in the area over the last few years.
The use of real-world data and real-world evidence reflects a general shift away from a drug development paradigm involving large RCTs with thousands of patients. We need to understand how medicines affect the actual population beyond a narrow clinical trial setting, and real-world evidence helps bridge that gap.
How has the use of RWD and RWE shifted with COVID?
We have seen a paradigm shift because of COVID. For example, there has been provision made for GPs and hospitals to exchange data on patients to help with disease management and monitoring. We have also seen an enormous vaccine surveillance effort in the UK reviewing who has had the vaccine and what is happening with adverse effects. While vaccine surveillance is not new, it is an unprecedented large scale effort in a condensed timeframe.
It was crucial that early on in the pandemic, authorities advised against using therapies off-label unless they were in a clinical trial such as RECOVERY because of the need to acquire evidence on their efficacy.
The trials were simplified to enrol as many patients as possible and examined a very defined endpoint.
The UK Vision for Clinical Research published last month aims to allow much greater participation in trials for patients and healthcare professionals with options from randomised controlled studies with specialist endpoints to more pragmatic and observational research.
What steps will be needed for RWE to transform drug development in the future?
Technology will develop irrespective of the actions of the pharmaceutical industry. The future for RWE will rely on regulators coming to a position that supports pharma to actively harness the available data. We also need ongoing health data research and open discussion with the public about what they want to see in terms of systems. I also expect public-private collaborations such as the IMI Big data for better outcomes project to play an essential role in building a new future for RWE.
About Dr Sheuli Porkess
Dr Sheuli Porkess is the Director of Actaros Consultancy, a consultancy focusing on transforming the development and use of medicines.
Sheuli is an experienced pharmaceutical physician with industry expertise in medicine development, medical affairs and research policy within the UK and internationally, across multiple therapy areas. Sheuli is a Fellow of the Faculty of Pharmaceutical Medicine in the UK and a Global Fellow in Medicine Development with IFAPP.
Sheuli holds a number of roles including the Chair of the Policy and Communications Group at the Faculty of Pharmaceutical Medicine, membership of the Scientific Advisory Boards for C2-Ai, a digital health company, and the AGILE pandemic clinical trial platform, as well as being a member of the NIHR AI for Multiple Long-term conditions (AIM) Programme Steering Group and a member of the DHSC COVID Prophylaxis Candidate Prioritisation Group.